Clinical Scorecard: An Overview of Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Disorders: Insights and Future Directions from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT)

At a Glance

Key Highlights

• HSCT has become an integral and standard-of-care treatment for multiple sclerosis and systemic sclerosis, representing around 80% of transplants for autoimmune diseases.
• Autologous HSCT involves mobilization, collection, and reinfusion of hematopoietic stem cells, often with conditioning regimens including lymphodepleting serotherapy such as anti-thymocyte globulin.
• Immune reconstitution post-HSCT shows renewal of naïve B cells, regulatory B and T cells, and re-diversification of T-cell receptor repertoire, potentially resolving autoimmune-mediated immune disturbances.

Guideline-Based Recommendations

Diagnosis

• Careful patient selection balancing benefits and risks of HSCT versus alternative treatments.
• Use of multidisciplinary teams including HSCT and disease specialists for assessment.

Management

• Use of autologous HSCT with peripheral blood stem cells mobilized by G-CSF with or without cyclophosphamide.
• Conditioning regimens vary by disease and center; intensity tailored to patient and disease characteristics.
• Consider graft manipulation such as CD34 selection and ex vivo T cell depletion to remove self-reactive lymphocytes.

Monitoring & Follow-up

• Long-term follow-up to assess immune reconstitution and disease remission.
• Registry data collection through EBMT-ADWP for outcome tracking.

Risks

• Treatment-related morbidity including infections, infertility, and organ damage.
• Risks vary with conditioning intensity and prior treatments; reduced intensity conditioning may reduce morbidity.

Patient & Prescribing Data

Patients with severe, refractory autoimmune diseases primarily multiple sclerosis, systemic sclerosis, and Crohn’s disease undergoing autologous HSCT.

HSCT is a one-off procedure aiming for durable remission; treatment intensity and conditioning regimens are individualized; registry data support improved outcomes with center experience and multidisciplinary care.

Clinical Best Practices

• Employ a multidisciplinary approach integrating HSCT and autoimmune disease specialists.
• Utilize peripheral blood stem cells with cryopreservation for graft collection.
• Tailor conditioning regimens to disease indication and patient factors to balance efficacy and toxicity.
• Monitor immune reconstitution markers such as naïve B cells and regulatory lymphocyte populations post-transplant.
• Contribute data to and utilize EBMT-ADWP registry for benchmarking and research.

References

• European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party